Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome
editing company, today announced new safety and efficacy data in 7
patients with transfusion-dependent beta thalassemia (TDT) treated
with renizgamglogene autogedtemcel (reni-cel; formerly known as
EDIT-301) in the Phase 1/2 EdiTHAL clinical trial. Reni-cel, the
first investigational AsCas12a gene-edited cell therapy medicine,
is being studied in the EdiTHAL trial as a potential one-time,
durable gene editing medicine for people living with TDT. The data
will be presented in a poster presentation at the European
Hematology Association (EHA) Hybrid Congress in Madrid, Spain, on
Friday, June 14 at 6:00 p.m. CEST (Noon EDT).
In the EdiTHAL trial to date, reni-cel was well-tolerated and
continues to demonstrate a safety profile consistent with
myeloablative conditioning with busulfan and autologous
hematopoietic stem cell transplant by all patients (N=7). Following
treatment with reni-cel, all EdiTHAL patients had early and robust
increase of total hemoglobin (Hb) and fetal hemoglobin (HbF) and
remain transfusion-free at last follow-up for a range of 4.1 to
12.8 months (N=7). All patients in the EdiTHAL trial underwent 1.0
apheresis and mobilization cycle.
“We continue to make significant progress in the development of
reni-cel for transfusion-dependent beta thalassemia. In these new
data at EHA, we demonstrate that all patients experienced early
increases in fetal hemoglobin, maintained hemoglobin levels above
the transfusion threshold and are free from red blood cell
transfusions following reni-cel infusion. These data further
support our belief that reni-cel has the potential to be a
clinically differentiated, one-time, durable medicine that can
provide life-changing clinical benefits to patients,” said Baisong
Mei, M.D., Ph.D., Chief Medical Officer, Editas Medicine. “I would
like to thank the participants, their families and caregivers,
clinicians, and colleagues at collaborating institutions that
contribute to the EdiTHAL trial.”
“The preliminary safety and efficacy results from the EdiTHAL
trial demonstrate this investigational medicine has been
well-tolerated and shows promising efficacy for patients. Reni-cel
has the potential to be a functional cure for people living with
transfusion-dependent beta thalassemia, and we look forward to
continuing to evaluate its effectiveness for these patients,” said
Haydar Frangoul, M.D., M.S., Medical Director, Pediatric Hematology
and Oncology, Sarah Cannon Research Institute and HCA Healthcare’s
TriStar Centennial Children’s Hospital.
Efficacy of reni-cel in Patients with
Transfusion-dependent Beta ThalassemiaIn the EdiTHAL
trial, patients demonstrated early and robust total Hb and HbF
increases, with total Hb rising above the transfusion independence
threshold of 9.0 g/dL. For patients with ≥6 months follow-up, the
mean (standard deviation; SD) total Hb and HbF concentrations at
month 6 were 12.1 g/dL (1.3 g/dL) and 10.9 g/dL (1.5 g/dL) (n=6),
respectively, which were sustained at or above the transfusion
threshold from 6 months through subsequent follow-ups.
All patients (N=7) have been transfusion free for a range of 4.1
to 12.8 months after receiving the last red blood cell transfusion
at 0.5–2.2 months post-reni-cel infusion.
All patients in the EdiTHAL trial showed sustained high levels
of editing in the HBG1 and HBG2 promoter regions.
Safety of reni-cel in Patients with
Transfusion-dependent Beta ThalassemiaReni-cel was
well-tolerated and demonstrated a safety profile consistent with
myeloablative conditioning with busulfan and autologous
hematopoietic stem cell transplant by all evaluated EdiTHAL trial
patients (N=7).
After reni-cel infusion, all patients (N=7) demonstrated
successful neutrophil and platelet engraftment. Neutrophil
engraftment occurred at a median of 23 days (min: 16 days, max: 30
days), and platelet engraftment occurred at a median of 38 days
(min: 24 days, max: 49 days).
No serious adverse events (SAEs) related to reni-cel treatment
in the EdiTHAL trial have been reported.
EHA PresentationsIn addition to the EdiTHAL
poster presentation, Editas will also present data from the RUBY
clinical trial of reni-cel for the treatment of severe sickle cell
disease in an oral presentation on Saturday, June 15.
EdiTHAL Poster Presentation Details:
Title: Reni-cel, the first AsCas12a gene-edited
cell therapy, shows promising preliminary results in key clinical
outcomes in transfusion-dependent beta thalassemia patients treated
in the EdiThal trialPresenting Author: Haydar
Frangoul, M.D., M.S., Medical Director, Sarah Cannon Pediatric
Hematology/Oncology & Cellular Therapy at TriStar Centennial,
Nashville, TN, United StatesDate/Time: Friday,
June 14, 2024, 6:00 – 7:00 p.m. CEST / Noon – 1:00 p.m.
EDTLocation: Hall 7, IFEMA MADRID Recinto
Ferial (Fairgrounds)Session: Poster Session
RUBY Oral Presentation Details:
Title: Reni-cel, the first AsCas12a gene-edited
cell therapy, led to hemoglobin normalization and increased fetal
hemoglobin in severe sickle cell disease patients in an interim
analysis of the RUBY trialPresenting Author: Rabi
Hanna, M.D., Department of Pediatric Hematology Oncology and Blood
and Marrow Transplantation, Cleveland Clinic Children’s, Cleveland,
OH, United StatesDate/Time: Saturday, June 15,
2024, 11:30 a.m. – 12:45 p.m. CEST/ 5:30 – 6:45 a.m.
EDTLocation: Hall Velasquez, IFEMA
MADRID Recinto Ferial (Fairgrounds)Session:
s425 Gene therapy, cellular immunotherapy and vaccination –
Clinical
The abstracts can be accessed on the EHA website and the
presentations can be accessed on the Editas Medicine website in the
posters and presentations section.
Reni-cel is currently being investigated in a clinical study in
patients with severe sickle cell disease (RUBY trial, NCT04853576)
and transfusion-dependent beta thalassemia (EDITHAL trial,
NCT05444894). In addition to the clinical data update from the RUBY
trial and the EdiTHAL trial at EHA, the Company will present a
further clinical update from both trials by year-end 2024.
About renizgamglogene autogedtemcel
(reni-cel)Reni-cel, formerly known as EDIT-301, is an
experimental gene editing medicine under investigation for the
treatment of severe sickle cell disease (SCD) and
transfusion-dependent beta thalassemia (TDT). Reni-cel consists of
patient-derived CD34+ hematopoietic stem and progenitor cells
edited at the gamma globin gene (HBG1 and HBG2) promoters, where
naturally occurring fetal hemoglobin (HbF) inducing mutations
reside, by AsCas12a, a novel, proprietary, highly efficient, and
specific gene editing nuclease. Red blood cells derived from
reni-cel CD34+ cells demonstrate a sustained increase in fetal
hemoglobin production, which has the potential to provide a
one-time, durable treatment benefit for people living with severe
SCD and TDT.
About the EdiTHAL TrialThe EdiTHAL trial is a
single-arm, open label, multi-center Phase 1/2 study designed to
assess the safety and efficacy of reni-cel in patients with
transfusion-dependent beta thalassemia. Patients will receive a
single administration of reni-cel. Additional details are available
on www.clinicaltrials.gov (NCT05444894).
About the RUBY TrialThe RUBY trial is a
single-arm, open-label, multi-center Phase 1/2/3 study designed to
assess the safety and efficacy of reni-cel in patients with severe
sickle cell disease. Enrolled patients will receive a single
administration of reni-cel. The RUBY trial marks the first time
AsCas12a was used to successfully edit human cells in a clinical
trial. Additional details are available
on www.clinicaltrials.gov (NCT04853576).
About Editas MedicineAs a
clinical-stage gene editing company, Editas Medicine is focused on
translating the power and potential of the CRISPR/Cas12a and
CRISPR/Cas9 genome editing systems into a robust pipeline of
treatments for people living with serious diseases around the
world. Editas Medicine aims to discover, develop, manufacture, and
commercialize transformative, durable, precision genomic medicines
for a broad class of diseases. Editas Medicine is the exclusive
licensee of Broad Institute’s Cas12a patent estate and Broad
Institute and Harvard University’s Cas9 patent estates for human
medicines. For the latest information and scientific presentations,
please visit www.editasmedicine.com.
Forward-Looking Statements This press release
contains forward-looking statements and information within the
meaning of The Private Securities Litigation Reform Act of 1995.
The words "anticipate," "believe,"
"continue," "could," "estimate,"
"expect," "intend," "may," "plan," "potential," "predict," "project," "target,"
"should," "would," and similar expressions are intended to identify
forward-looking statements, although not all forward-looking
statements contain these identifying words. Forward-looking
statements in this press release include statements regarding the
timing for the Company’s receipt and presentation of data from its
clinical trials, including presenting additional clinical data from
the RUBY and EdiTHAL trials by year-end 2024, and the potential of,
and expectations for, the Company’s product candidates. The Company
may not actually achieve the plans, intentions, or expectations
disclosed in these forward-looking statements, and you should not
place undue reliance on these forward-looking statements. Actual
results or events could differ materially from the plans,
intentions and expectations disclosed in these forward-looking
statements as a result of various important factors, including:
uncertainties inherent in the initiation and completion of clinical
trials, including the RUBY and EdiTHAL trials, and clinical
development of the Company’s product candidates, including
reni-cel; availability and timing of results from clinical trials;
whether interim results from a clinical trial will be predictive of
the final results of the trial or the results of future trials;
expectations for regulatory approvals to conduct trials or to
market products and availability of funding sufficient for the
Company’s foreseeable and unforeseeable operating expenses and
capital expenditure requirements. These and other risks are
described in greater detail under the caption “Risk Factors”
included in the Company’s most recent Annual Report on Form 10-K,
which is on file with the Securities and Exchange
Commission, as updated by the Company’s subsequent filings
with the Securities and Exchange Commission, and in other filings
that the Company may make with the Securities and Exchange
Commission in the future. Any forward-looking statements contained
in this press release speak only as of the date hereof, and the
Company expressly disclaims any obligation to update any
forward-looking statements, whether because of new information,
future events or otherwise.
Media and Investor Contact:
Cristi Barnett
(617) 401-0113
cristi.barnett@editasmed.com
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